The U.S. Food & Drug Administration (FDA) has granted Breakthrough Therapy status to Takeda Pharmaceuticals’ investigational, oral proteasome inhibitor, ixazomib (MLN9708), for the treatment of relapsed or refractory systemic light-chain amyloidosis. This is the first proteasome inhibitor and first investigational therapy for systemic light-chain amyloidosis to receive Breakthrough Therapy designation.

Systemic light-chain amyloidosis is a rare and aggressive protein misfolding disorder with fewer than 3,000 cases diagnosed in the United States every year. It is characterized by the deposition of amyloid in bodily organs and tissues. While systemic light-chain amyloidosis can affect different organs in different people, it frequently affects the heart, kidneys, liver, spleen, nervous system, and gastrointestinal tract. There are no approved treatments in the United States or globally for systemic light-chain amyloidosis, representing a significant unmet medical need.

The development program for ixazomib in this indication progressed directly from a phase I to a phase III clinical trial, TOURMALINE-AL1, which is currently evaluating ixazomib plus dexamethasone in patients with relapsed or refractory systemic light-chain amyloidosis. This is the only phase III trial for relapsed or refractory systemic light-chain amyloidosis and it is recruiting globally. ■